Introduction <p>Managing patients with highly frequent seizures poses significant challenges for clinicians due to their high resistance to therapy. This study aims to evaluate the 12-month efficacy, safety, and tolerability of PER as the sole add-on therapy for patients with highly active epilepsy in a real-world setting.</p> Methods <p>Data from the previous Italian retrospective, observational, multicenter “PERampanel as Only Concomitant Antiseizure Medication” (PEROC) study were analyzed, categorizing patients by baseline seizure frequency into three groups: &lt; 5, 5–20, and &gt; 20 seizures/month. Retention, responder (≥ 50% seizure reduction) rates, seizure-free rates and adverse events (AEs) were analyzed. Sub-analyses examined early (≤ 1 previous ASM) vs. late (&gt; 2 previous ASMs) add-on groups.</p> Results <p>The sample included 485 patients with focal and generalized epilepsy: 354 with &lt; 5 seizures/month, 79 with 5–20, and 52 with &gt; 20 seizures/month. Retention rates at 12&#xa0;months were 75.1%, 68%, and 58.1.7%, respectively. Perampanel significantly reduced seizure frequency in all groups, with responder rates of 71.2%, 61.8%, and 63.2% at the 12-month follow-up. Patients with more frequent seizures (&gt; 20 and 5–20 seizures/month) had lower seizure-free rates (15.8% and 23.5%) compared to those with &lt; 5 seizures/month (49.5%, <i>p</i> = 0.001). AEs, mainly dizziness and irritability occurred in 30% of patients, without significant differences between groups (<i>p</i> = 0.092).</p> Conclusions <p>PER, as the sole adjunctive therapy, demonstrated good effectiveness and tolerability in a real-world setting, even for patients with highly active epilepsy. These findings suggest PER as a valuable early treatment option to improve seizure control and quality of life in this challenging population.</p>

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Effectiveness of perampanel as only concomitant antiseizure medication for highly active epilepsy: insight from a real-world, multicenter retrospective study

  • Angelo Pascarella,
  • Marilisa Pasquale,
  • Domenico Abelardo,
  • Sara Gasparini,
  • Oreste Marsico,
  • Roberta Cutellè,
  • Vittoria Cianci,
  • Alfonso Iudice,
  • Francesca Bisulli,
  • Paolo Bonanni,
  • Emanuele Caggia,
  • Alfredo D’Aniello,
  • Carlo Di Bonaventura,
  • Jacopo C. DiFrancesco,
  • Elisabetta Domina,
  • Fedele Dono,
  • Antonio Gambardella,
  • Carla Marini,
  • Alfonso Marrelli,
  • Sara Matricardi,
  • Alessandra Morano,
  • Francesco Paladin,
  • Rosaria Renna,
  • Marta Piccioli,
  • Pasquale Striano,
  • Michele Ascoli,
  • Angela La Neve,
  • Emilio Le Piane,
  • Alessandro Orsini,
  • Claudia Torino,
  • Simone Beretta,
  • Umberto Aguglia,
  • Edoardo Ferlazzo,
  • Carla Arbasino,
  • Irene Bagnasco,
  • Pierangelo Barbero,
  • Emanuele Bartolini,
  • Maria A. Bassetti,
  • Vincenzo Belcastro,
  • Vanni Boero,
  • Alice Bonuccelli,
  • Alessandro Bulgari,
  • Emanuele Caggia,
  • Roberto Cantello,
  • Susanna Casellato,
  • Natascia Casula,
  • Edward Cesnik,
  • Clotilde Ciampa,
  • Anna Maria Cipriani,
  • Mario Coletti Moja,
  • Marta Conti,
  • Giangennaro Coppola,
  • Francesco Corea,
  • Giovanni Crichiutti,
  • Giuseppe D’Orsi,
  • Filippo Dainese,
  • Alberto Danieli,
  • Marco De Curtis,
  • Valentina De Giorgis,
  • Luigi Del Gaudio,
  • Francesco Deleo,
  • Giancarlo Di Gennaro,
  • Giacomo Evangelista,
  • Francesco Fortunato,
  • Stefania Filipponi,
  • Attilio Gagliano,
  • Thea Giacomini,
  • Francesca Gilio,
  • Alfonso Giordano,
  • Loretta Giuliano,
  • Shalom Haggiag,
  • Stella Jensen,
  • Angelo Labate,
  • Laura Licchetta,
  • Angelica Lupato,
  • Greta Macorig,
  • Anna Mammì,
  • Maria Margherita Mancardi,
  • Daniela Marino,
  • Barbara Mostacci,
  • Lorenzo Muccioli,
  • Susanna Negrin,
  • Francesca Operto,
  • Alessandro Orsini,
  • Pasquale Palumbo,
  • Mariagrazia Pascarella,
  • Alessia Peretti,
  • Gabriella Perri,
  • Giuseppina Pustorino,
  • Federica Ranzato,
  • Antonella Riva,
  • Serena Servo,
  • Ilaria Sammarra,
  • Laura Siri,
  • Orazio Spitaleri,
  • Andrea Stabile,
  • Gionata Strigaro,
  • Costanza Varesio

摘要

Introduction

Managing patients with highly frequent seizures poses significant challenges for clinicians due to their high resistance to therapy. This study aims to evaluate the 12-month efficacy, safety, and tolerability of PER as the sole add-on therapy for patients with highly active epilepsy in a real-world setting.

Methods

Data from the previous Italian retrospective, observational, multicenter “PERampanel as Only Concomitant Antiseizure Medication” (PEROC) study were analyzed, categorizing patients by baseline seizure frequency into three groups: < 5, 5–20, and > 20 seizures/month. Retention, responder (≥ 50% seizure reduction) rates, seizure-free rates and adverse events (AEs) were analyzed. Sub-analyses examined early (≤ 1 previous ASM) vs. late (> 2 previous ASMs) add-on groups.

Results

The sample included 485 patients with focal and generalized epilepsy: 354 with < 5 seizures/month, 79 with 5–20, and 52 with > 20 seizures/month. Retention rates at 12 months were 75.1%, 68%, and 58.1.7%, respectively. Perampanel significantly reduced seizure frequency in all groups, with responder rates of 71.2%, 61.8%, and 63.2% at the 12-month follow-up. Patients with more frequent seizures (> 20 and 5–20 seizures/month) had lower seizure-free rates (15.8% and 23.5%) compared to those with < 5 seizures/month (49.5%, p = 0.001). AEs, mainly dizziness and irritability occurred in 30% of patients, without significant differences between groups (p = 0.092).

Conclusions

PER, as the sole adjunctive therapy, demonstrated good effectiveness and tolerability in a real-world setting, even for patients with highly active epilepsy. These findings suggest PER as a valuable early treatment option to improve seizure control and quality of life in this challenging population.